What are clinical trials?

You may have heard of the term “evidence-based medicine.” This means that any therapy, medication, or intervention that a doctor recommends should have evidence that supports that decision. Evidence is created through scientific research, including trials where patients are given new treatments so scientists can study the results. Clinical trials can be designed to answer questions such as: Is this medication or device safe to use in humans? Will this newly developed therapy lead to better patient outcomes than the current standard? Are there any side effects to a new therapy? Clinical trials play a vital role in producing reliable and accurate data that helps inform physicians’ decisions about patient care. And they offer new options for men with prostateThe prostate is a walnut-sized gland located between the bla... Full Definition cancerCancer is a group of diseases where cells grow abnormally an... Full Definition.

Clinical trials are safe.

Clinical trials aren’t wild experiments. Patient safety and privacy is of the highest priority during clinical trials. There is rigorous scientific and legal oversight that a clinical trial must follow, all with the goal of protecting patients from harm. Before a clinical trial begins, the study design is submitted to an Institutional Review Board (IRB) that includes doctors, researchers, and administrators who make sure the planned study is ethical and correctly designed to answer an important scientific question. Another governing body is the Food and Drug Administration (FDA), which monitors and approves clinical trials at every step. If it becomes clear that a device or medical therapy is causing harm to the patients in the study, the trial will be stopped immediately. Alternatively, sometimes a new experimental therapy works so well that the trial is stopped because it is no longer ethical for other patients not to receive the newer and more effective therapy.

How does something become a clinical trial?

All clinical trials begin with an idea, or hypothesis, about a new treatment that may improve the welfare and life span of patients with a specific disease. These ideas can come from a laboratory or from clinicians and researchers who work directly with patients. First, the group generates a scientific question, or hypothesis. Next, they must develop a project protocol that explains exactly how their study will answer the hypothesis. After rigorous review by multiple governing agencies, the protocol may be accepted.

Clinical trials in Phase I involve a small group of people who are closely monitored for tolerance of a new treatment and any potential side effects. If a drug is being tested, typically the dose will start very small and the goal is to monitor the patient’s well-being. If the drug is tolerated, then the next group of people participating in the trial will receive a slightly higher dose. This continues until the best dose of the drug is determined.

In Phase II the focus becomes whether the new treatment works well or not. Here, a small group of people, typically at one academic center, receive the treatment. If the results are promising, the clinical trial may move into Phase III, which involves a much larger study group usually located at multiple academic centers. With the increase in size, the study provides enough data to create evidence that there is a significant benefit to the new treatment.

Some studies are randomized. This means the study patients are divided into two groups, with one group considered the “control group” and the other group considered the “experimental group.” The control group might receive the standard of care or perhaps even a placebo treatment, while the experimental group receives the new treatment that is being studied. The patients in these groups are randomized—neither the patients nor the doctors choose which group a patient is placed in—so that characteristics such as age, medical conditions, and more are equally represented in both groups. Additionally, the people who analyze the study results are generally “blinded,” which means they receive “de-identified” patient information and data. Analyzing the data this way minimizes the potential for human bias.

Why clinical trials are good for science

It wasn’t until the 19th century that medicine began systematically adopting scientific method to test and apply new medical and surgical therapies to treat disease. By adhering to standard scientific practices and evidence-based medicine, there was an unprecedented period of progress and sharing of new scientific discoveries. The same is true today, and a randomized controlled clinical trial can be considered the gold standard for advancing the practice of medicine. There is no other study design that provides such valuable data that has the power to change clinical practice in short periods of time.

Why clinical trials are good for you

Deciding to participate in a clinical trial for prostate cancer treatment is a personal decision that every patient must make for himself. The benefits of participating include access to new therapies that have not been made widely available yet, close monitoring by a multidisciplinary health team, and the satisfaction of contributing to a body of knowledge that will help others.

All patients have a right to “informed consent.” Informed consent means that your doctor or part of your healthcare team will explain in specific terms the risks and benefits of participating in a clinical trial. Such a discussion is an important opportunity to ask questions and discuss aspects of the clinical trial with your doctor. Be sure you understand the overarching goal of the study, the direct risk/benefit ratio for you as an individual, and what kind of extra testing or procedures you may undergo. Your doctor and care team will only proceed after you express good understanding of the steps involved. If you choose to join the trial, you will be helping patients everywhere, including yourself!